Both demand for and investment in fill/finish capacity continue to rise.

Focus on Fill and Finish

There are many benefits of employing a reformulation strategy, but companies must also overcome a variety of challenges too.

Reformulating Success

The industry revisits the pros and cons of microbial fermentation at scale for biotherapeutics.

Weighing the Benefits of Fermentation for New Biotherapies

BFS technology can help maintain sterility during the biologics manufacturing process.

Improving Sterility Using Blow-Fill-Seal Technology

A simplified downstream process can save time and costs but requires enabling technologies.

Considerations for Simplifying Downstream Processes

Using a minimum valuable product and process approach would make it is possible to discipline and structure the development of biopharmaceuticals in the fastest way possible.

Introducing a New Concept for Value-Driven Acceleration in Biopharma Development

Awareness of recently implemented—or ongoing—advances by the pharmacopoeias can help biotherapeutic manufacturers remain compliant with current requirements.

Biologic Standards in the Pharmacopoeias: An Update

 Washington Editor, jillwechsler7@gmail.com.

Regulatory authorities and biopharma companies are engaged in a range of collaborations to harmonize research methods, approval standards, and quality production methods key to expanding global drug production. While some initiatives predated the COVID-19 pandemic, the ongoing health emergency has elevated the importance of collaboration to speed biomedical research efforts and to ensure broad access to quality medicines and vaccines.

An important message from COVID-19 is the critical need for global regulatory convergence, commented Peter Marks, director of the Center for Biologics Evaluation and Research (CBER), at the PDA–FDA Joint Regulatory Conference held on Sept. 12, 2022. He warned, though, that the move toward greater information sharing seen in recent years is “slipping away” as the pandemic recedes, speaking to the importance of joint efforts to provide pharmacovigilance information and review findings.

Such initiatives are not new for FDA, which has worked for decades to provide technical assistance to regulatory authorities in low- and middle-income countries to advance regulatory convergence and improve drug production methods. The Promoting Quality of Medicines Plus (PQM+) program implemented by the US Pharmacopeia with support from the US Agency for International Development (USAID) aims to build in-country regulatory capacity to ensure broad production of high-quality treatments for neglected tropical diseases, tuberculosis, and other infections. A FDA online seminar explained these issues for global regulators from lower-income regions, with agency experts describing the agency’s submission and regulatory processes and collaborative efforts to advance drug quality testing (1).

Pharmaceutical manufacturers in Europe and other industrial nations similarly are working to ensure equitable access to vaccines and treatments in future pandemics, as seen in a framework devised by the International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) to improve responses to future health emergencies. A declaration issued in July 2022 calls for the assessment and identification of successes in pathogen surveillance and vaccine development and distribution during the pandemic. The organization calls on biopharmaceutical companies to help regulators strengthen pandemic preparedness and response capabilities to accelerate the delivery of vaccines, treatments, and diagnostics in their regions (2).

Similarly, the International Coalition of Medicines Regulatory Authorities (ICMRA) aims to harmonize standards for collecting real-world evidence, for assessing manufacturing information, and for conducting inspections to support quality assessments. One program encourages collaborative assessment of chemistry, manufacturing, and control (CMC) data to facilitate post-approval changes and inspections in different regions (3).

FDA also supports global clinical testing and review efforts, such as Project Orbis at its Oncology Center of Excellence (OCE), which was launched in 2019 to advance cancer drug development and regulation. Under the program, sponsors submit applications for high-impact oncology products simultaneously to participating regulators in Australia, Canada, Singapore, Switzerland, Brazil, the United Kingdom, and the United States to facilitate collaboration and agreement on reviews for new cancer drugs and supplements for added indications. While OCE coordinates the program, each regulatory authority makes independent regulatory decisions.

The International Council for Harmonisation (ICH) continues to seek agreement from regulatory authorities and industry on new and revised standards for clinical testing and quality manufacturing of drugs and biologics. Current efforts aim to advance common strategies for validation and selection of analytical procedures in drug testing, for assessment and control of impurities in drugs, and for validating bioanalytical assays. A new initiative is developing guidelines for including pregnant and breastfeeding women in clinical trials, while another project is mapping harmonized approaches for regulating generic drug development (4). These and other ICH initiatives will be further discussed at its annual assembly Nov. 15–16, 2002 in Korea.

One obstacle to global biomedical regulatory harmonization, however, arises from an effort in the European Union to revise and strengthen its data privacy framework, despite the potential for blocking regulatory access to necessary clinical trial information and adverse event reports. The EU’s General Data Protection Regulations (GDPR) adopted in 2018 aim to limit the release of individual information related to a range of personal activities, including health care. Unfortunately, these policies have created difficulties for FDA bioresearch monitoring program (BIMO) inspections and for sponsors submitting information on study subjects in new drug applications and other documents.

A report from FDA’s Europe Office posted Aug. 9, 2022 describes how the GDPR prevents FDA BIMO investigators from reviewing and copying clinical health data involving EU citizens participating in multi-national clinical trails and in reviewing certain demographic and other information normally included in applications sent to the agency (5). While the EU data framework aims to protect private information on a range of activities, the resulting limits on FDA’s ability to assess EU health information pose difficulties in advancing a global clinical research framework and common regulatory policies.

Global Access to Medicines, Including Anti-TB Medicines

IFPMA, “Pharma Proposes to Work With G7 and G20 on a Joint Solution for Better Access to Vaccines and Treatments Around the World for Future Pandemics,” News Release, July 19, 2022.

ICMRA, “International Pilot Programmes to Streamline Regulatory Assessments and Inspections–Call For Industry Applications,” Press Release, June 2022.

ICH, “ICH Assembly Meeting, Athens, Greece, May 2022 A Successful Hybrid Meeting,” Press Release, June 1, 2022.

H. Messick, “How a European Data Law Is Impacting FDA,” 


Volume 35, Number 10
October 2022
Pages: 8-9

When referring to this article, please cite it as J. Wechsler, “Advancing Global Collaboration for Drug Testing, Regulation, and Manufacturing,” 

Articles

FDA backs joint reviews, common research policies, and modern production methods around the world.

Advancing Global Collaboration for Drug Testing, Regulation, and Manufacturing

Chris Spivey is the editorial director of 

In business, understanding future key variables ahead of competitors provides a decisive advantage. In highly regulated industries, such as pharmaceuticals, this includes policy and compliance filing changes. Despite an editors’ mission being to educate and inform, I have been conflicted about sharing the following. The reason the first and second rules of fight club are famously “do not talk about fight club” is that productive events often depend on a delicate framework balancing expression, fun, and flexible social mores against a need for certainty and durable authoritative institutions. The annual PDA/FDA Joint Regulatory Conference is, in this way, the same as fight club. I concluded that sharing the fact that there are secrets is entirely different from sharing any of the secrets themselves.

Advertising for the conference says it “is designed to share approaches that facilitate sustainable compliance with CGMPs [current good manufacturing practices] by addressing emerging risks to manufacturing and supply chain reliability [and that] it will provide forums for open discussion between FDA representatives and industry experts, and offer opportunities for the application of approaches that enhance assurance of raw material quality, quality assurance management of contract manufacturing relationships, root cause analysis, and use of current technologies... [and that] key themes will include leveraging the digital and automation revolution, contamination control strategy, latest FDA CGMP trends, how to respond effectively to FDA 483s” and so on (1).

Pivotally, this description left out the secret that “open discussion” extends to a willingness to listen and take into account new information and anecdote, genuine attempts to learn about and resolve short and long-term problems, and heartfelt honest opinion and conjecture. Cumulatively, that makes this event easily the most useful time spent during the year. Details themselves are expressly “off-the-record” and not to be talked about outside of the event. That’s the engine that generates the power of this event, creating more light than heat. Attendance will provide a decisive advantage to anyone attending, and there’s clearly no reason to fight about that.

1. FDA, 2022 PDA/FDA Joint Regulatory Conference, Events, 

When referring to this article, please cite it as C. Spivey, "Industry’s Best Secret," 

One can only learn the secrets of "fight club" by attending its meetings.

Industry’s Best Secret

Susan J. Schniepp is a fellow at Regulatory Compliance Associates, Inc. and a member of BioPharm International's Editorial Advisory Board.

Q: I just attended a meeting where someone talked about the concept of real compliance. Can you explain what they were referring to?

A: This is a great question because I believe there is a difference between regulatory compliance and real compliance. Let’s start with what we mean by regulatory compliance. The current good manufacturing practice (CGMP) regulations for drugs contain minimum requirements for the methods, facilities, and controls used in the manufacturing, processing, labeling, and packaging of drug products.

In the United States, some of the laws are delineated in section 501(a)(2)(B) of the Food, Drug, and Cosmetic Act, which states, “A drug shall be deemed to be adulterated if the methods used in, or the facilities or controls used for, its manufacture, processing, packaging, or holding do not conform to or are not operated or administered in conformity with current good manufacturing practice to assure that such drug meets the requirements of this [Act] as to safety and has the identity and strength, and meets the quality and purity characteristics, which it purports or is represented to possess” (1).

) Part 211 establishes the regulations based on the law. The regulations contained in 21 CFR 211 do not tell you how to specifically meet these regulatory requirements but, rather, tell you what is expected of your quality management system in order to achieve compliance to the regulations. The goal of regulatory compliance is to satisfy the regulations to achieve a satisfactory inspectional outcome.

Real compliance is how a company interprets the regulatory requirements and expectations and applies them to their specific operations. Real compliance is rooted in a commitment to quality at all levels of an organization. A real compliance approach takes a high-level view of quality and allows the organization to put in controls and oversight that helps prevent deviations from occurring. It also provides a better understanding of the impact of a deviation when they do occur during manufacturing. Organizations that adopt a quality-based philosophy (real compliance) minimize the impact when deviations of a serious nature occur. Real compliance is the practical application of a quality management system and how it is integrated in the facility. The goal of real compliance is to achieve a sustainable state of control at the facility based on scientific principles, best practices, and continual improvement efforts. The best way to explain the difference between regulatory compliance and real compliance is to perform a thorough compare-and-contrast exercise.

The regulations clearly require companies to have written procedures that are in the form of standard operating procedures (SOPs). The common language used in 21 

 211 to indicate SOPs are required can be exemplified by 21 

 211.22 “Responsibilities of quality control unit”, which states “(d) The responsibilities and procedures applicable to the quality control unit shall be in writing; such written procedures shall be followed” (2). This type of wording can be found is sections of 21 

 211 governing personnel qualifications, building and facilities, equipment, production and process control, labeling issuance, warehousing procedures, etc. The regulatory requirement is met by having SOPs, but real compliance is met having SOPs that reflect your operations. For example, if you outsource product testing you would not need an SOP on how to investigate out-of-specification (OOS) results because you are not performing that activity. Instead, you might replace it with an SOP describing how you work with your contract test laboratory when they are investigating an OOS result associated with your product. Another example between regulatory compliance and real compliance is just having a complaint department vs. having a complaint department that is manned by employees that understand their responsibilities and are trained in complaint handling best practices. Another comparison demonstrating the difference between regulatory compliance and real compliance is having an organization chart that separates quality and operations vs. having that same organization chart supported by SOPs that demonstrate quality has the responsibility and authority to operate independently.

Here’s a more practical example of regulatory compliance vs. real compliance. US 21 

 211.25(a) requires “Each person engaged in the manufacture, processing, packing, or holding of a drug product shall have education, training, and experience or any combination thereof, to enable that person to perform the assigned functions” (2). A regulatory compliance approach would be to offer every employee annual GMP training and document their attendance. A real compliance approach would be to provide effective training to the employees by assessing their comprehension of the material and offering follow-up training as necessary.

Another practical example of regulatory compliance vs. real compliance is examining how a company approaches investigations. A strictly regulatory compliance approach to an investigation results in a firm not determining the true root cause of an issue because the focus is on completing the investigation in a self-imposed time frame instead of focusing on the quality of the investigation. Real compliance organizations focus on determining the true root cause(s) and using that information across their manufacturing facility to drive continuous improvements and prevent recurrence of the deviation that prompted the investigation.

Taking a regulatory compliance only approach to operations may be less expensive in the short term, but in the long term it will most likely result in more expenditure to fix the problems that could have been avoided if a real compliance approach was implemented throughout
the organization.

US Code, Federal Food, Drug, and Cosmetic Act,
Section 501(a)(2)(B).

Susan J. Schniepp is distinguished fellow at Regulatory Compliance Associates.


Volume 35, Number 10
October 2022
Pages 54, 52

When referring to this article, please cite it as S.J. Schniepp, “Regulatory Compliance Versus Real Compliance,” 

Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, addresses the difference between regulatory compliance and real compliance.

Regulatory Compliance Versus Real Compliance

Mike Hennessy Jr. is the President and CEO of 

Post-approval changes (PACs) are inextricable sequelae of living in the real world. “They are inevitable and necessary throughout the life of a drug product—to implement new knowledge, maintain a state of control, and drive continual improvement … Many PACs require regulatory agency approval by individual countries before implementation. Because of the global regulatory complexity, individual PACs usually take years for full worldwide approval even when they reduce patient risk, improve compliance, or enhance the manufacturing process or test methods” (1).

A modification to a vaccine, sold in a dozen different countries, can require more than 100 new approvals. If successfully handled by a robust internal (and already well proven) pharmaceutical quality system (PQS) to manage these modifications, time, energy, and money—for both the manufacturer and also the regulators—would be saved. Both 

 have published a series of position papers on this topic. They cover “Change of Lot or Extension of Shelf-life of Reference Standard”, “Change in Size of Thermal Shipping Solution Used for Transport of Product”, and “Addition of a Testing Lab to an Existing Testing Site,” among other facets.

The 2022 Parenteral Drug Association (PDA)-FDA Joint Regulatory Conference in Washington, DC truly showcased how well industry stakeholders can communicate and cooperate. Sufficient trust has been established, one would think, such that some of these product modifications can be managed in a streamlined and expedited fashion. Particularly since these positions are proposed by senior quality leaders (chief quality officers and heads of quality) from more than 20 global pharmaceutical companies, and “are speaking with “One-Voice-Of-Quality” (1VQ) to advocate for an effective management of specific PACs that currently are handled as a prior-approval change in some countries, but where a standard science and risk-based approach concludes that these should be downgraded to a notification or handled only in the PQS (2).

Special mention should be given to Emma Ramnarine, Global Head, External Development Collaborations at Genentech/Roche, and Anders Vinther, VP Quality at Kronos Bio, for galvanizing action in this field.

Pharmaceutical Regulatory Science Team, Industry One-Voice-of Quality (1VQ) for Post-Approval Changes (PAC), accessed Sept. 26, 2022. 

When referring to this article, please cite it as M. Hennessy, "Optimizing Quality," 

The 1VQ papers advocate for the idea that certain post-approval changes should be streamlined and expedited.

Optimizing Quality

Citius Pharmaceuticals, a late-state biopharmaceutical company, recently announced a collaboration with Haider Madhi, MD, an assistant professor at the University of Pittsburgh. The collaboration will focus on an investigator-initiated trial evaluating I/ONTAK (denileukin diftitox), a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments, in combination with pembrolizumab, for the treatment of recurrent or metastatic solid tumors.

According to a company press release, the trial is an open label, Phase I/Ib study designed to evaluate regulatory T-cell (T-regs) cell depletion through the denileukin diftitox and pembrolizumab combination; it consists of two parts. Part I is a dose escalation study of four cohorts (3, 6, 9, 12 mcg of denileukin diftitox) that is expected to enroll 18–30 patients. Part II is a dose expansion study enrolling approximately 40 patients to evaluate the safety and tolerability of the recommended dose. Additionally, the study will investigate the alteration of the immune microenvironment within tumors and peripheral blood.

"This study will expand the body of knowledge about [denileukin diftitox] unique mechanism-of-action targeting the CD25 component of the IL2-receptor, which is present on both malignant T-cells (T-cell leukemias and lymphomas) and immunosuppressive T-regs," said Myron Czuczman, chief medical officer, Citius Pharmaceuticals, in the release. "Preclinical research in a syngeneic solid tumor mouse model shows that [denileukin diftitox] enhances anti-tumor activity and significantly extends [the] survival benefit of anti-PD-1 [programmed cell death 1] therapy. This data provides a positive signal of denileukin diftitox's potential in the immuno-oncology space.”

"Encouraging clinical data emerging in the field of tumor immunotherapy have demonstrated that therapies focused on enhancing T-cell responses against cancer result in a significant survival benefit in patients with advanced malignancies,” said Mahdi in the release. “Overexpression of PD-L1 [programmed cell death ligand 1] on tumor cells has been reported to impede anti-tumor immunity, resulting in immune evasion. The interruption of the PD-1:PD-L1 pathway combined with diminishing the suppressive effect by T-regs may represent an attractive strategy for restoring tumor-specific T-cell immunity.”

In addition to this study withUniversity of Pittsburgh, Citius is collaborating with an investigator-initiated study at the University of Minnesota. This Phase I dose-finding study is to evaluate denileukin diftitox prior to tisagenlecleucel chimeric antigen receptor-T cell therapy in patients with diffuse large B-cell lymphoma; the study enrolled its first patient in May 2021.

Citius and the University of Pittsburgh will investigate I/ONTAK in combination with pembrolizumab for treatment of recurrent or metastatic solid tumors.

Citius Pharmaceuticals Announces Clinical Collaboration with the University of Pittsburgh

Forge Biologics, a contract development and manufacturing organization focused on gene therapies, announced the addition of plasmid DNA manufacturing to its Columbus, Ohio, facility on Sept. 27, 2022. The addition is designed to complement the company’s existing manufacturing services for gene therapy programs, including anti-adeno-virus process development, analytic development, current good manufacturing practice (CGMP) manufacturing, and automated final fill capabilities.

According to a company press release, the plasmid manufacturing services encompass three grades: research-grade, for discovery, research, and development; GMP-pathway, for early stage clinical trial CGMP adeno-associated virus (AAV) manufacturing; and CGMP, for late-stage clinical and commercial CGMP manufacturing. Research-grade and GMP-pathway plasmid grade manufacting is currently available, and CGMP plasmid production is expected to come online in 2023.

“The launch of plasmid DNA manufacturing enables clients to better streamline the production of their AAV programs by vertically integrating plasmids into our manufacturing process,” said John Maslowski, chief commercial officer, Forge Therapeutics, in the release. “The ability to streamline vendor management, better control timeline risks, and accelerate end-to-end AAV manufacturing are important differentiators when upscaling to our 5000-L [C]GMP bioreactors for clients and patients.”

Forge Biologics will offer three grades of plasmid manufacturing services at its Columbus, Ohio, location.

Forge Biologics Launches Plasmid DNA Manufacturing Services

PerkinElmer announced on Sept. 26, 2022 that it has launched the Cellaca PLX Image Cytometry System, a platform designed to enable researchers to assess multiple Critical Quality Attributes (CQAs) of cell samples in a single automated workflow; this includes cell identity, quality, and quantity.

The system combines image cytometer hardware, software, validated consumables, and trackable data reporting; it is designed to eliminate complex calibration procedures or intense training requirements. According to a company press release, the new offering will provide researchers expanded cell sample CQA analysis options beyond flow cytometry and staining methods, which have historically required a variety of different instruments and analytical methods. By combining these capabilities, researchers can detect multiple markers simultaneously and perform immunophenotyping and viability assays in seconds.

"Pharmaceutical companies have invested heavily in cell and gene therapy, but they struggle to assess the complex cell samples required to meet immense scientific demands and regulatory rigor across their research and manufacturing processes," said Alan Fletcher, senior vice-president, Life Sciences, PerkinElmer, in the release. "While the Cellaca PLX Image Cytometer platform is therapeutic area agnostic, it is expected to be especially beneficial for researchers working in CAR-T cell therapy who want to streamline their phenotyping of immune cells for downstream processes."

PerkinElmer has unveiled a cell analysis solution to streamline cell and gene therapy research and manufacturing. 

PerkinElmer Unveils Cell Analysis Solution

Ten years ago, FDA launched an initiative to expand the role of patients in informing regulators and sponsors on most harmful effects of a disease and primary goals of treatment that can lead to desirable medical approaches. To further advance and refine the process, FDA is highlighting how the Patient-Focused Drug Development (PFDD) program has encouraged active involvement of many patient groups in bringing their perspectives into the process of developing, testing, and approving new medicines.

The primary goal of PFDD is to “better incorporate the patient’s voice in drug development and evaluation,” says Theresa Mullin, associate director for strategic initiatives in the Center for Drug Evaluation and Research (CDER), who has led this effort since its establishment. The initial program featured FDA-led meetings but was expanded to include a wider range of sessions organized by patient groups themselves, Mullin explained in a webinar sponsored by the 

The PFDD initiative was established by legislation reauthorizing the 

 (PDUFA) in 2012. It called for FDA to organize 20 meetings each year to discuss patient experiences, which symptoms have the most significant impact on daily life, and how well current therapies treat those symptoms. The program grew to 24 meetings in the first year as more groups recognized the benefits of hearing patient perspectives on disease severity and on potential effects of treatment. Then in 2016, PFDD further expanded to include discussions organized by patient groups. There have been 55 of these externally led meetings, which follow the format of the agency-organized meetings in being open to the public and producing detailed reports to post on the FDA website.

Every one of these meetings “was very powerful,” Mullin commented, noting that physicians at FDA said that they had never heard these perspectives and that reviewers found that patient comments helpful in assessing the risks and benefits of a new therapy. And even though patients did not advocate for any particular therapies, they discussed the impact of available treatments and where new therapy could have an impact, a process that supported the development of new treatments for sickle cell disease complications.

To further improve the PFDD process, FDA has issued a series of guidance documents to advance the reliability of PFDD tools and methods. Two final guidances address how to 

collect comprehensive and representative input

how to assess which aspects of disease burden and treatment

outlines processes for selecting, developing, or modifying clinical outcome assessments. Still to come is a fourth guidance on incorporating clinical outcome assessments into endpoints to support regulatory decision making. These advisories aim to encourage manufacturers and researchers to utilize high-quality methods for collecting patient experience information that will be useful in regulatory decisions and to move away from multiple differing efforts for developing clinical assessment measures and tools that repeat the PFDD process with every manufacturer.

In looking to the future, Mullin envisions some kind of “national catalog” of standard core clinical outcomes and assessments based on patient perspectives. This would provide reliable measures of clinical outcomes for disease areas that are meaningful to patients and could gain support from FDA’s Standard Core Clinical Outcome Assessments Grant Program. The overarching aim is to ensure that clinical trial protocols are reasonable and accessible for patients and that clinical outcome assessment measures compare things that people care about.

FDA is highlighting how the Patient-Focused Drug Development program has encouraged active involvement of many patient groups in bringing their perspectives into the process of developing, testing, and approving new medicines.

FDA Expands Patient Input on Drug Development 

The Massachusetts Justice department announced on Sept. 26, 2022 that Biogen had agreed to pay approximately $900 million to resolve allegations concerning submissions of false claims to Medicare and Medicaid. The suit, filed and litigated by former Biogen employee, Michael Bawduniak, under the whistleblower provisions of the False Claims Act, alleged that the company paid kickbacks to physicians to induce them to prescribe Biogen drugs.

According to the department’s press release, the suit alleges that from Jan. 1, 2009, through March 18, 2014, Biogen held programs through which it offered and paid remuneration, such as speaker and consulting fees, to health care professionals to induce them to prescribe three of their drugs: Avonex (interferon beta-1a), Tysabri (natalizumab), and Tecfidera (dimethyl fumarate). These actions would be a violation of the federal Anti-Kickback Statute, which prohibits exchange (or offer to exchange) anything of value to induce or reward the referral of business reimbursable by federal healthcare programs (1).

“Bawduniak doggedly pursued this matter on behalf of the United States for over seven years,” said General Brian M. Boynton, principal deputy assistant attorney and head of the Department of Justice’s Civil Division, in the release. “The settlement announced today underscores the critical role that whistleblowers and their attorneys play in utilizing the False Claims Act to combat fraud affecting federal healthcare programs.”

Under the terms of the settlement, Biogen will pay approximately $844 million to the US and approximately $56 million to 15 states. The US will pay Bawduniak a share of the federal recovery. The claims resolved by the settlement are allegations only and there has been no determination of liability.

United States Department of Justice, District of Massachusetts

1. American Society of Anaesthesiologists, “Anti-Kickback Statute and Physician Self-Referral Laws (Stark Laws),” 

Biogen has agreed to pay $900 million to settle federal and state claims that it paid kickbacks to physicians to encourage them to prescribe its drugs.

Biogen to Settle Kickbacks Lawsuit for $900 Million

The National Institute of Health (NIH) recently unveiled a new process for revealing key details of choroideremia, a rare genetic disorder that leads to blindness. Found through a study by the National Eye Institute (NEI), the approach combines traditional eye imaging techniques with adaptive optics, a type of technology that enhances imaging resolution.

According to an agency press release from Sept. 13, 2022, this approach was developed by Johnny Tam, head of the Clinical and Translational Imaging Unit at NEI. By combining adaptive optics with indocyanine green dye, the team was able to view live cells in the retina, including light-sensing photoreceptors, retinal pigment epithelium (RPE), and choroidal blood vessels. This allowed the researchers to see the extent to which choroideremia disrupts these tissues, information which can be used to design effective treatments for the condition.

The major findings of the study concerned the retinal pigment epithelium (RPE), a layer of pigmented cells needed for the nourishment and survival of photoreceptors. Patients with choroideremia had RPE cells that were significantly enlarged, with some enlarged as much as five-fold; by contrast, photoreceptor and blood vessel layers had less disruptions. According to the release, this suggested to the researchers that RPE disruptions may play a significant role in the disease.

“It’s not obvious at first, but using an existing tool in the clinic, we can monitor and track the cellular status of the RPE layer,” said Tam in the release. “This could prove valuable in identifying which patients would benefit the most from therapeutic interventions.”

A combination of eye imaging techniques and adaptive optics has revealed key details of choroideremia.

Novel Imaging Approach Reveals Key Details About Rare Eye Disease

Actylis, a global manufacturer and sourcing expert of raw materials and performance ingredients, announced its debut on Sept. 26, 2022. The new company combines Aceto and 10 industry specialists into an integrated global ingredient powerhouse; A&C, A&C Bio Buffer, Biotron Laboratories, Cascade Chemistry, Finar, Inter-Actifs, IsleChem, Pharma Waldhof, Syntor Fine Chemicals, and Talus.

According to a company press release, Actylis aims to address the major unmet need for better and more dependable access to critical raw materials and performance ingredients needed for the manufacture of highly regulated products in key industries. The combination of the many specialists will enable Actylis’ hybrid manufacturing and sourcing model, which is designed to provide various benefits to its customers in high-growth end markets.

“Actylis unites multiple industry specialists with a wide range of capabilities into a new, global enterprise with a unique hybrid approach that is greater than the sum of its parts,” said Gilles Cottier, CEO of Actylis, in the release. “This consolidation enables us to offer customers across diverse locations and industries highly flexible, customized solutions addressing their specific needs, while assuring reliable on-time delivery of the high-quality ingredients essential to their success.”

Actylis combines Aceto and its 10 acquired specialists into a singular global brand.

Actylis Debuts as an Integrated Global Specialty Ingredients Manufacturing and Sourcing Provider

Octagon Therapeutics, a preclinical-stage biotechnology company, announced a partnership with Novo Nordisk on Sept. 20, 2022. The research collaboration will use Octagon’s functional target discovery approach and novel chemistry strategy in disease areas in which Novo Nordisk has specific expertise.

The program selectively targets a population of pathologically activated B cells that contribute to autoimmune diseases like systemic lupus and vasculitis. According to a company press release, this approach can be applied in other disease areas to target disease-driving processes while retaining healthy immune functioning. A recent pilot project, initiated through Novo Nordisk’s Co-creation Greenhouse Accelerator program, demonstrated proof-of-concept in identifying atypical characteristics of other immune cells during disease; this ultimately led to the discovery of new therapeutic targets.

"Octagon has a unique platform technology to look into functional biology in a novel way.It makes use of clinical samples and primary immune cells to identify new drug targets,” said Uli Stilz, vice-president, Bio Innovation Hub, Novo Nordisk, in the press release. “Combined with our disease understanding in the cardiometabolic space and Octagon´s approach in targeting specific lymphocyte populations that drive disease progression, it will be exciting to see what therapeutic discoveries the collaboration can lead to."

"Novo Nordisk has a strong expertise in diabetes and related cardiometabolic conditions like [nonalcoholic fatty liver disease] and kidney disease." said Isaac Stoner, CEO, Octagon Therapeutics, in the press release."This collaboration will enable Octagon to expand our technology into these disease areas and to further leverage the impressive clinical sample collection that Novo Nordisk has available."

The collaboration will use Octagon’s functional target discovery approach and novel chemistry strategy in disease areas in which Novo Nordisk has specific expertise.